Efficacy and Safety of Avacopan in patients with ANCA-associated vasculitis: A Systematic Review and Meta-analysis

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Background Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) is a rare autoimmune vasculitis characterized by inflammation of small- to medium-sized blood vessels. While conventional treatments such as glucocorticoids and immunosuppressant improve disease control, they carry a high burden of toxicity. Avacopan, an oral complement 5a receptor inhibitor, has emerged as a potential steroid-sparing therapy. This meta-analysis aimed to evaluate the efficacy and safety of avacopan in patients with AAV. Methods This systematic review and meta-analysis followed PRISMA guidelines. A total of seven randomized controlled trials involving patients with AAV were included. Primary outcomes assessed included clinical efficacy (remission at 26 and 52 weeks, relapse, and vasculitis worsening), glucocorticoid toxicity, safety and tolerability, and organ-specific adverse effects. Pooled odds ratios (ORs) or mean differences (MDs) with 95% confidence intervals (CIs) were calculated. Results Avacopan demonstrated significantly higher remission rates at 52 weeks (OR 1.79; 95% CI: 1.18–2.72; p = 0.006) and lower relapse rates (OR 0.39; 95% CI: 0.21–0.72; p = 0.003) compared to standard care. Steroid-related toxicity was significantly reduced, with lower GTI-AIS and cumulative worsening scores at 13 and 26 weeks. Notably, avacopan reduced infection rates (OR 0.69; p = 0.02) and adverse psychiatric events (OR 0.55; p = 0.03). While other adverse events were numerically lower, they were not statistically significant, indicating a comparable or improved safety profile. Conclusion Avacopan improves long-term clinical outcomes and reduces glucocorticoid-related toxicity in AAV, representing a safer, effective adjunct to standard therapy.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.